This week: FDA has placed regulatory brakes on an autologous, gene-corrected cell therapy for epidermolysis bullosa and six new UK modules for cell therapy manufacturing have been completed.
The news highlights:
Mallinckrodt (Staines-upon-Thames, UK) have announced positive results of their pivotal Phase III clinical trial evaluating the safety and efficacy of StrataGraft® — their novel, investigational regenerative tissue treatment for patients with deep, partial-thickness thermal burns. Both co-primary trial endpoints were successfully met and, as a result, Mallinckrodt plans to submit a Biologics License Application for StrataGraft to the US FDA.
James H. Holmes IV, co-lead study investigator and Director of Wake Forest Baptist Medical Center’s Burn Center (NC, USA), commented: “Treatment advances are needed that can help minimize or eliminate the need to harvest skin tissue for autografting, as the second wound created by removing healthy skin can be associated with complications and can be even more painful than the burn wound itself. The positive top-line results of the Phase III trial suggest that this investigational regenerative tissue, if approved, could provide burn surgeons with an alternative treatment option for deep partial-thickness burns.”
Abeona Therapeutics (TX, USA) has received a clinical hold letter from the US FDA detailing that FDA approval will not be granted for the company’s planned Phase III clinical trial investigating the use of EB-101 — an investigational, autologous, gene-corrected cell therapy — in patients with recessive dystrophic epidermolysis bullosa. Before approval is granted, the FDA has requested that Abeona Therapeutics submit further data concerning the transport stability of EB-101.
JoÃ£o Siffert, CEO of Abeona, stated: “Initiating the VIITALâ„¢ pivotal Phase III trial for EB-101 is the Company’s top priority. Efforts to gather supplemental data points on transport stability of EB-101 are ongoing and we are confident that the requested additional data will be submitted to the FDA promptly. We remain deeply committed to advancing EB-101 to provide a desperately needed treatment for RDEB patients.”
Cell and Gene Therapy Catapult (CGT Catapult; London, UK) have announced the completion of the construction of six new manufacturing modules at their Stevenage (UK) base. Each module can accommodate the parallel processing of up to 20 autologous cell or bioreactor processes. The successful construction effectively doubles CGT Catapult’s cell therapy manufacturing capabilities.
Keith Thompson, CEO of CGT Catapult, stated: “We are very pleased to be able to double the existing capacity of the CGT Catapult manufacturing center…The growth of this center is integral to the success of the industry in the UK, and we can now collaborate potentially with up to a further six companies to support them in developing their GMP manufacturing processes, preparing for large scale and commercial supply.”
For more weekly cell therapy news, read previous editions of the cell therapy weekly.